From Patient, To Science, To Solution

We discover and develop medicines to help improve human health. Today’s evolving scientific knowledge and advancing technical capabilities are providing unprecedented opportunities to match unmet patient need with breakthrough innovation.

 

Nowhere is this more evident than in the discovery story behind our new FDA approved medicine for women with postmenopausal osteoporosis. It’s a tale of inspiration from an extraordinary place, and how we turned a genetic discovery into a new medicine – it’s a journey from patient to science to solution.

 

This is a story involving NASA, mice in space and nearly three decades of research. It began with research that identified a new gene, which turned out to be the underlying cause of a genetic condition that results in bone overgrowth – sclerosteosis.

 

At first glance, osteoporosis and sclerosteosis couldn’t seem more different since osteoporosis patients have bones that become weak and brittle while sclerosteosis patients don’t produce sclerostin and their bones are thicker and stronger than normal. 
 

 

As sclerosteosis is the opposite of osteoporosis, our scientists hypothesized that the defective gene in sclerosteosis patients could possibly encode a novel regulator of bone formation, amenable to pharmaceutical manipulation, which could lead to a potential new drug target for treating low bone mass disorders such as osteoporosis. 
 

 

By combining reverse engineering and genomics our scientists were able to turn a genetic discovery into a new medicine.